Phase 1 Study of BLU-285 in Patients with GIST v1
Research type
Research Study
Full title
A Phase 1 Study of BLU-285 in Patients with Gastrointestinal Stromal Tumors(GIST) and other Relapsed and Refractory Solid Tumors.
IRAS ID
184105
Contact name
Robin Jones
Contact email
Sponsor organisation
Blueprint Medicines Corporation
Eudract number
2015-001660-18
Clinicaltrials.gov Identifier
Duration of Study in the UK
4 years, 2 months, 1 days
Research summary
Research Summary
This study is a first-in-human study that will test an experimental drug called BLU-285 when it is taken orally by patients with Gastrointestinal Stromal Tumours (GIST) or other solid tumours.
GIST are a rare form of tumour which may develop at any point along the gastrointestinal (GI) tract and are associated with a mutation in either of two particular genes known as KIT and PDGFRα.
Surgery is the most common treatment for patients with GIST and chemotherapy and radiotherapy don’t work well for GIST; therefore researchers have been looking for other ways to treat GIST.
The study drug, BLU-285, operates by interfering with factors thought to promote the growth of certain gastrointestinal cancer cells.
The study is in two part and aims to establish the most effective dose of the experimental drug with the least side effects (Part 1), and confirm that the chosen dose of the study drug is safe and well tolerated (Part 2).Eligible patients will undergo blood and urine sample collection, vital signs measurement, ECG monitoring, disease-response assessment by magnetic resonance imaging (MRI) or computed tomography (CT) and adverse event collection. Continuous Holter monitoring will be performed in patients in Part 2 of the study at selected centres only.
Approximately 60 adult patients (≥ 18 years of age) will be enroled in the study, which will be conducted at study centres in Europe, the US and Asia. Patients will participate in the study for a minimum of approximately 3 months. The expected duration of the study is approximately 50 months.
The study is sponsored by Blueprint Medicines Corporation.
Summary of Results
If no, explain why they haven't: In this study, patient data sharing responsibility was transferred to site Investigators and staff, based on local regulations & guidelines. The sponsor did not share any data directly with patients. The overall study results leading to the approval of avapritinib in the select indications are available publicly. The results are also posted by the sponsor on clinicaltrials.gov and will be posted on EudraCT.REC name
South Central - Berkshire B Research Ethics Committee
REC reference
15/SC/0552
Date of REC Opinion
9 Oct 2015
REC opinion
Further Information Favourable Opinion